PMID- 35179251 OWN - NLM STAT- MEDLINE DCOM- 20220505 LR - 20220731 IS - 1096-8652 (Electronic) IS - 0361-8609 (Print) IS - 0361-8609 (Linking) VI - 97 IP - 6 DP - 2022 Jun 1 TI - Fostamatinib for the treatment of warm antibody autoimmune hemolytic anemia: Phase 2, multicenter, open-label study. PG - 691-699 LID - 10.1002/ajh.26508 [doi] AB - Patients with relapsed warm antibody autoimmune hemolytic anemia (wAIHA) have limited treatment options. Fostamatinib is a potent, orally administered spleen tyrosine kinase inhibitor approved in the United States and Europe for the treatment of adults with chronic immune thrombocytopenia (ITP). This phase 2 study evaluated the response to fostamatinib, administered at 150 mg BID orally with or without food in adults with wAIHA and active hemolysis with hemoglobin (Hgb) <10 g/dL who had failed at least one prior treatment. Hemoglobin levels and safety assessments were performed at visits every 2 weeks. The primary endpoint was Hgb >10 g/dL with an increase of >/=2 g/dL from baseline by week 24 without rescue therapy or red blood cell transfusion. Eleven of 24 (46%) patients achieved the primary endpoint. Increases in median Hgb were detected at week 2 and sustained over time. Median lactate dehydrogenase levels and reticulocyte counts generally declined over time with little change in median haptoglobin levels. The most common adverse events (AEs) were diarrhea (42%), fatigue (42%), hypertension (27%), dizziness (27%), and insomnia (23%). AEs were manageable and consistent with the fostamatinib safety database of over 3900 patients across multiple diseases (rheumatoid arthritis, B-cell lymphoma, COVID-19, and ITP). No new safety signals were detected. Fostamatinib may be a promising therapeutic option for wAIHA. A randomized, double-blind, phase 3 study is nearing completion. CI - (c) 2022 The Authors. American Journal of Hematology published by Wiley Periodicals LLC. FAU - Kuter, David J AU - Kuter DJ AUID- ORCID: 0000-0003-2084-8810 AD - Division of Hematology, Massachusetts General Hospital, Boston, Massachusetts, USA. FAU - Rogers, Kerry A AU - Rogers KA AD - Division of Hematology, Department of Medicine, The Ohio State University, Columbus, Ohio, USA. FAU - Boxer, Michael A AU - Boxer MA AD - Division of Hematology-Oncology, Arizona Oncology, Tucson, Arizona, USA. FAU - Choi, Michael AU - Choi M AD - Moores Cancer Center, University of California San Diego, La Jolla, California, USA. FAU - Agajanian, Richy AU - Agajanian R AD - Division of Hematology-Oncology, The Oncology Institute of Hope and Innovation, Downey, California, USA. FAU - Arnold, Donald M AU - Arnold DM AD - Department of Medicine, Michael G. DeGroote School of Medicine, McMaster University, Hamilton, Ontario, Canada. FAU - Broome, Catherine M AU - Broome CM AUID- ORCID: 0000-0003-1507-2851 AD - Lombardi Cancer Center, Division of Hematology, MedStar Georgetown University Hospital, Washington, District of Columbia, USA. FAU - Field, Joshua J AU - Field JJ AD - Blood Center of Wisconsin, Medical College of Wisconsin, Milwaukee, Wisconsin, USA. FAU - Murakhovskaya, Irina AU - Murakhovskaya I AD - Department of Medicine (Hematology), Albert Einstein College of Medicine, Bronx, New York, USA. FAU - Numerof, Robert AU - Numerof R AD - Development and Medical Affairs, Rigel Pharmaceuticals, Inc, South San Francisco, California, USA. FAU - Tong, Sandra AU - Tong S AD - Development and Medical Affairs, Rigel Pharmaceuticals, Inc, South San Francisco, California, USA. LA - eng SI - ClinicalTrials.gov/NCT02612558 PT - Clinical Trial, Phase II PT - Journal Article PT - Multicenter Study DEP - 20220303 PL - United States TA - Am J Hematol JT - American journal of hematology JID - 7610369 RN - 0 (Aminopyridines) RN - 0 (Morpholines) RN - 0 (Oxazines) RN - 0 (Pyridines) RN - 0 (Pyrimidines) RN - SQ8A3S5101 (fostamatinib) SB - IM MH - Adult MH - Aminopyridines MH - *Anemia, Hemolytic, Autoimmune/drug therapy MH - *COVID-19 MH - Humans MH - Morpholines MH - Oxazines MH - Pyridines MH - Pyrimidines PMC - PMC9313871 COIS- David J. Kuter has received research funding from Actelion (Syntimmune), Agios, Alnylam, Amgen, Argenx, Bristol Myers Squibb (BMS), Immunovant, Kezar, Principia, Protalex, Rigel, and Takeda (Bioverativ) UCB; and has served in a consulting role for Actelion (Syntimmune), Agios, Alnylam, Amgen, Argenx, Bristol Myers Squibb (BMS), Caremark, CRICO, Daiichi Sankyo, Dova, Genzyme, Immunovant, Incyte, Kyowa-Kirin, Merck Sharp Dohme, Momenta, Novartis, Pfizer, Platelet Disorder Support Association, Principia, Protalex, Protalix, Rigel, Sanofi, Genzyme, Shionogi, Shire, Takeda (Bioverativ), UCB, Up-To-Date, and Zafgen. Kerry A. Rogers has received research funding from Genentech, AbbVie, Novartis, and Janssen; has served in a consulting role for Acerta Pharma, AbbVie, Genentech, Pharmacyclics, AstraZeneca, and Innate Pharma; and has received travel funding from AstraZeneca. Michael A. Boxer has served on speaker's bureaus for Sanofi and Pharmacosmos. Michael Choi has received research funding from Pharmacyclics, Rigel, Sunesis, TG Therapeutics, and Velosbio. Donald Arnold has received research funding from Novartis and BMS; and has served in a consulting role for Rigel, Novartis, Amgen, UCB, and Principia. Catherine M. Broome has served in a consulting role for Alexion, Argenx, Sanofi, and Appelis. Robert Numerof and Sandra Tong are employees of Rigel Pharmaceuticals. The remaining authors declare no competing financial interests. EDAT- 2022/02/19 06:00 MHDA- 2022/05/06 06:00 PMCR- 2022/07/25 CRDT- 2022/02/18 08:41 PHST- 2022/02/11 00:00 [revised] PHST- 2021/10/06 00:00 [received] PHST- 2022/02/15 00:00 [accepted] PHST- 2022/02/19 06:00 [pubmed] PHST- 2022/05/06 06:00 [medline] PHST- 2022/02/18 08:41 [entrez] PHST- 2022/07/25 00:00 [pmc-release] AID - AJH26508 [pii] AID - 10.1002/ajh.26508 [doi] PST - ppublish SO - Am J Hematol. 2022 Jun 1;97(6):691-699. doi: 10.1002/ajh.26508. Epub 2022 Mar 3.